Z Factor Limited is a University of Cambridge spin-out company founded by Jim Huntington, Professor of Molecular Haemostasis at the Cambridge Institute for Medical Research.
The Z Factor team is engaged in the discovery of new drugs to treat Alpha-1-Antitrypsin Deficiency (AATD) which is a significant cause of liver and lung disease, resulting from a defect in the gene encoding Alpha-1-antitrypsin. Individuals with two defective copies of the gene, making up around 1 in 2000 of the Western population, typically develop emphysema starting in their 30s. They are also at an increased risk of developing liver diseases such as cirrhosis and cancer. Around 2% of people have one defective copy of this gene, and are at a 5-fold increased risk of developing Chronic Obstructive Pulmonary Disease (COPD) as they age.
The most common mutation causing AATD is called the Z mutation, which disrupts the normal folding of the protein. Professor Huntington and his team at University of Cambridge obtained the crystallographic structure of this mutant form of Alpha-1-antitrypsin, which allowed for the first time the rational design of drugs that could correct folding and prevent the development of associated diseases.
Z Factor has identified dozens of molecules that can correct the folding defect caused by the Z mutation, and shown that some of these drug candidates can increase Alpha-1-antitrypsin levels in an in vivo model of AATD. In 2017 Z Factor raised £7 million in a Series A round led by Medicxi and in which CIC participated.The company is now working to select the best molecules for use as a drug in human trials, with the lead candidate expected to reach the clinic in 2019.
Z Factor is based on the Babraham Research Campus, just south of Cambridge, UK. It operates as a virtual biotechnology company using an established network of out-sourced drug discovery and development providers co-located on the Babraham campus.